Abstract: Disclosed is a pharmaceutical composition for the prevention or treatment of brain cancer, containing a compound capable of penetrating the blood-brain barrier as an active ingredient, the pharmaceutical composition for the prevention or treatment of brain cancer is capable of efficiently penetrating the blood-brain barrier of the brain to thereby effectively inhibit LRRK2 protein expression, and is thus useful in the prevention or treatment of brain cancer.

Abstract: The present invention relates to complexes of nocardamine (desferrioxamine E) with at least one metal, specifically with Zn2+ or Ga3+ or silver or gold or any combinations thereof. The invention further provides procedures for the preparation of the complexes and pharmaceutical compositions comprising said complexes or any combinations of said complexes, as well as methods using the compositions and complexes of the invention in modulating cytokine levels in a subject and thereby treating inflammation-associated disorders.

Abstract: Provided is a method of treatment of COVID-19 and/or preventing infection with SARS-CoV-2 comprising: administering to an individual in need thereof a therapeutically effective dose of mifepristone, or a pharmaceutically acceptable salt and/or analogue thereof, and a therapeutically effective dose of amlodipine, or a pharmaceutically acceptable salt thereof.

Abstract: Disclosed herein, in certain embodiments, are compositions, solutions, and soft gelatin capsules comprising 17-alpha hydroxyprogesterone caproate (17-HPC). In certain embodiments, also disclosed herein are methods, dosing regimens, and kits for use in the treatment of a disease or condition.

Abstract: The disclosure provides an injectable neurosteroid nanoparticle formulation comprising nanoparticles having a D50 of less than 2000 nm the nanoparticles comprising a neurosteroid of Formula I, where the variables R1-R9 and X are defined herein and at least one surface stabilizer. The surface stabilizer can be a polymeric surface stabilizer such as hydroxyethyl starch, dextran, or povidone. The injectable neurosteroid nanoparticle formulation can be an intravenous formulation. The disclosure also provides a lyophilized powder of the injectable neurosteroid nanoparticle formulation that can be reconstituted in an aqueous solution prior to administration. The disclosure provides injectable neurosteroid nanoparticle formulations and dry powders of such formulations that have been sterilized by ebeam irradiation.

Abstract: Methods of treating non-alcoholic steatohepatitis (NASH) are provided. For instance, the methods comprise administering 5-cholesten-3,25-diol, 3-sulfate (25HC3S) ora salt thereof.

Abstract: Methods of treating non-alcoholic steatohepatitis (NASH) are provided. For instance, the methods comprise administering 5-cholesten-3,25-diol, 3-sulfate (25HC3S) or a salt thereof.

Abstract: Cancer treatment methods including adjusting t to in vivo concentration of a selected metabolite in a patient to levels effective to activate selected metabolic pathways that promote differentiation of cancer cells. In some examples the methods for treating cancer include adjusting the in vivo concentration of one or more of vitamin-D, niacin, ascorbic acid, ascorbyl palmitate, and butyrate.

Abstract: Mitochondrially targeted antioxidants are used to prevent and treat severe inflammatory conditions including viral infection (such as COVID-19), systemic shock, trauma, burns, surgery associated with significant tissue damage, toxic damage, and damage associated with autoimmune reactions.

Abstract: Disclosed herein are composition and methods of treating a condition where enhanced immunogenicity is desired. Some embodiments disclosed herein relate to compositions comprising a T-cell activator and/or proliferator, one or more immune checkpoint inhibitor, and a FPPS inhibitor. Some embodiments relate to methods of treating cancer by co-administering plinabulin, one or more immune checkpoint inhibitor, and a FPPS inhibitor to a subject in need thereof.

Abstract: A stable AST-3424 injection preparation, being a solution containing 0.1-200 mg/ml or 1-200 mg/ml of AST-3424 active pharmaceutical ingredient. A solvent in the solution contains a C2-C8 monohydric alcohol. The preparation method comprises the following steps: subjecting the AST-3424 active pharmaceutical ingredient and a partial prescription volume of ethanol to a first dissolution and formulation; adding a prescription volume of propylene glycol for a second dissolution and formulation; and then adding the remaining prescription volume of ethanol for mixing and dissolving to obtain a solution containing 1-200 mg/ml of AST-3424 active pharmaceutical ingredient. Provided is an AST-3424 injection for injection, wherein the solvent is water, a glucose solution, ethanol, or propylene glycol, the solutes consist of the AST-3424 active pharmaceutical ingredient, an isotonic regulator, and a pH regulator. The concentration of the AST-3424 active pharmaceutical ingredient of the injection is 0.001-1.

Abstract: Disclosed herein, inter alia, are methods of using thiosaccharide compounds for treating coronavirus infection.

Abstract: The invention relates to a nutrient composition, a food including the same and use of the nutrient composition. The nutrition composition includes: human milk oligosaccharide, preferably neutral fucosylated human milk oligosaccharide, which is preferably one or more selected from the group consisting of: 2?-fucosyl lactose, 3?-fucosyl lactose, lacto-N-fucopentaose I, lacto-N-difucohexaose I, lacto-N-difucohexaose II, preferably 2?-fucosyl lactose (2?-FL); milk fat globule membrane; and choline and/or an edible choline derivative. The nutritional composition can improve the brain development and intelligence of babies, infants and young children, especially promote neurodevelopment such as neuron maturation, synaptogenesis and myelination.

Abstract: The disclosure provides for immunomodulatory oligosaccharides, and uses thereof.

Abstract: Disclosed are methods for treating cystic fibrosis characterized by a class I nonsense mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene in a subject in need thereof. The method comprises administering to the subject a pharmaceutical composition comprising an effective amount of an inhibitor selected from the group consisting of a sodium glucose co-transporter (SGLT) inhibitor, a Na+/K+-ATPase inhibitor, an SGLT1/Na+/K+-ATPase dual inhibitor, and combinations thereof.

Abstract: Provided are a reelin and/or VEGF-C production and/or activation promoter including a compound represented by Chemical Formula 1 or a salt thereof as an active ingredient, and a skin external composition including the same. (In Chemical Formula 1, each substituent is as defined in the specification.

Abstract: Novel aminoglycosides, represented by Formulae (e.g., a compound of Formula A, B, I, I*, III or III*, including compounds represented by Formula Ia, I**, I*a, I*b, IIIa, III**, III*a and, III*b), as defined in the instant specification, designed to exhibit stop codon mutation readthrough activity, are provided. Also provided are pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic diseases and disorders, such as diseases and disorders associated with stop codon mutations.

Abstract: The present disclosure provides compositions and methods that inhibit the activity of microRNAs, for example miR-22.

Abstract: The invention provides a method for improving attention of a healthy human subject by administering an effective amount of citidine-5?-diphosphocholine or a salt thereof to a healthy human subject.

Abstract: Provided is a composition of antiviral agent for use in prophylaxis or treatment against pathogenic infection. The liposomal antiviral agent of the composition has a liposome and an antiviral agent entrapped in the liposome. The antiviral agent has been stably encapsulated in the liposome, and the resulting liposomal antiviral agent is proven to be stably aerosolized or nebulized for administration via the inhalation route to treat a subject in need thereof with reduced side effect.

Abstract: Deoxynucleotide prodrugs for treatment of diseases characterized by unbalanced nucleotide pools are provided herein.

Abstract: Deoxynucleotide prodrugs for treatment of diseases characterized by unbalanced nucleotide pools are provided herein.

Abstract: RNA encoding an immunogen is co-delivered to non-immune cells as the site of delivery and also to immune cells which infiltrate the site of delivery. The responses of these two cell types to the same delivered RNA lead to two different effects, which interact to produce a strong immune response against the immunogen. The non-immune cells translate the RNA and express the immunogen. Infiltrating immune cells respond to the RNA by expressing type I interferons and pro-inflammatory cytokines which produce a local adjuvant effect which acts on the immunogen-expressing non-immune cells to upregulate major histocompatibility complex expression, thereby increasing presentation of the translated protein to T cells. The effects on the immune and non-immune cells can be achieved by a single delivery of a single RNA e.g., by a single injection.

Abstract: The present invention provides, among other things, improved methods and pharmaceutical compositions for treating cystic fibrosis based on codon optimized mRNA encoding a wild-type Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein.

Abstract: This invention relates generally to pharmaceutical compositions and preparations of polyribonucleotides and circular polyribonucleotides and uses thereof.

Abstract: The present invention relates to an inhibitor of asparagine synthase for use for the treatment of a disorder characterized by renal and/or liver cyst formation and relative pharmaceutical composition.

Abstract: Disclosed herein are compositions and methods for modulating a macrolide antimicrobial, modulating an immune function, increasing the effectiveness of an antibiotic, and/or increasing the number and effectiveness of alveolar macrophages in treating a vertebrate animal. The method comprises feeding to the animal a feed composition comprising (1, 3), (1, 6), beta-glucans and optionally mannans to increase the number of alveolar macrophage cells, and administering a macrolide antibiotic to the animal.

Abstract: Described herein are a solution, composition, and kit of poly (acetyl, arginyl) glucosamine (PAAG), methods of making the solution, and method of treating wounds with the solution, the method comprising administering to a subject an effective amount of a solution comprising PAAG, wherein the PAAG when administered topically contacts the wound, thereby treating the wound.

Abstract: The invention provides compositions and methods for treatment of gastrointestinal bleeding, such as upper GI bleeding. In certain aspects, the invention provides topical formulations that stop bleeding in the GI tract by forming a gel when a polymer in the formulation exceeds a threshold level (optionally a pre-determined threshold level) for a property of the polymer of the formulation, which threshold level may be for example, a physical (e.g., temperature), chemical (e.g., pH), or temporal threshold level following contact with the affected tissue. In certain aspects, a combination of properties, such as a combination of physical (e.g., temperature), chemical (e.g., pH), or temporal threshold levels determines when the polymer in the formulation transitions from a liquid to a gel. Methods of using such compositions are also provided.

Abstract: The present invention relates generally to a method of treating infectious conjunctivitis by administering to an infected eye of a subject a composition comprising povidone-iodine and an excipient that reduces the ocular toxicity of the composition, wherein the composition does not include a steroidal anti-inflammatory drug or a non-steroidal anti-inflammatory drug.

Abstract: A use of micro- and nano-MgH2 compound particles in the inhibition of Leishmania infection and in the treatment of Leishmaniasis is provided. The micro- and nano-MgH2 compound particles can be used to prepare a pharmaceutical composition for inhibiting Leishmania or to prepare a pharmaceutical composition for treating a skin or mucosal ulcer or visceral damage caused by Leishmania infection. The present disclosure discovers for the first time that the micro- and nano-MgH2 compound particles can significantly reduce the number of Leishmania in macrophages and inhibit the proliferation of Leishmania, indicating a very prominent insecticidal inhibition effect. The micro- and nano-MgH2 compound particles of the present disclosure can quickly and effectively cure a skin ulcer and/or a mucosal ulcer caused by Leishmania and an impaired function of an internal organ (mainly liver and spleen) caused by Leishmania and have high biosafety and huge clinical application values.

Abstract: The present invention provides stabilized amorphous calcium carbonate (ACC) formulations, comprising ACC and a non-aqueous liquid carrier in which the ACC is dispersed. The present invention further provides cosmetic and pharmaceutical compositions comprising ACC.

Abstract: The present invention provides an epithelial cancer therapeutic agent with less side effects. In order to achieve the above object, an epithelial cancer therapeutic agent of the present invention includes: at least one material selected from the group consisting of oxo acid, oxo acid ion, and oxo acid salt, wherein the epithelial cancer therapeutic agent treats or prevents epithelial cancer.

Abstract: The present invention provides a method of treating frontotemporal dementia, or a childhood genetic neurodegenerative disease such as Ataxia Telangiectasia (A-T), or neurodegenerative diseases such as Parkinson's disease or neuropsychiatric diseases comprising administering to a subject in need thereof an effective amount of chlorite composition, such as sodium chlorite. The present invention thereby provides a method of modulating the immune system in a subject in need thereof. Described herein are methods of administration and treatment.

Abstract: The present disclosure relates to packaged eyelid scrubs as well as methods for making the packaged eyelid scrubs. The packaged eyelid scrubs include a pharmaceutical composition that includes hypochlorous acid and menthol, and a package that includes an opaque airless spray container filled with the pharmaceutical composition. Methods of cleansing an eyelid using the pharmaceutical compositions are also described herein.

Abstract: The present invention relates to novel microporous zirconium silicate compositions that are formulated to remove toxins, e.g. potassium ions, from the gastrointestinal tract at an elevated rate without causing undesirable side effects. The preferred formulations are designed avoid increase in pH of urine in patients and/or avoid potential entry of particles into the bloodstream of the patient. Also disclosed is a method for preparing high purity crystals of UZSi-9 exhibiting an enhanced level of potassium exchange capacity. These compositions are particularly useful in the therapeutic treatment of hyperkalemia.

Abstract: Disclosed herein is a pharmaceutical composition comprising tetraarsenic hexoxide as an active ingredient. Having excellent antiviral activity against coronaviruses, the pharmaceutical composition can be advantageously applied to the prevention or treatment of coronavirus diseases.

Abstract: Disclosed herein is a method for treating a subject having, or at risk of having, a cancer, comprising administering to the subject a therapeutically effective amount of a tumor membrane vesicle (TMV) and a metformin. In some embodiments, the TMV comprises a B7-1 and/or IL-12 molecule anchored to a lipid membrane (e.g., by a GPI anchor). In some embodiments, the methods can further comprise administering an immune checkpoint inhibitor. The methods are useful for reducing tumor size and metastasis, and in improving anti-tumor immune responses.

Abstract: Provided herein are antibodies that specifically bind to Fms-like tyrosine kinase 3 (FLT3), chimeric antigen receptors (CARs) that specifically bind to FLT3, and engineered immune cells expressing such CARs (e.g. FLT3-specific CAR-T cells). The invention also provides making such antibodies, CARs, and engineered immune cells. The invention also provides using such antibodies, CARs, and engineered immune cells, for example for the treatment of a condition associated with malignant cells expressing FLT3 (e.g., cancer).

Abstract: This disclosure provides a method for treating a subject afflicted with a cancer comprising administering to the subject a combination of therapeutically effective amounts of an isolated population of natural killer (NK) cells, preferably activated and expanded NK (NKAE) cells, and an antibody or an antigen-binding portion thereof that binds specifically to C-X-C Chemokine Receptor 4 (CXCR4) expressed on the surface of a cancer cell.

Abstract: This disclosure provides modified B cells which produce heterologous antibodies and co-express cargo proteins. The modified B cells may be stimulated by binding of a cognate antigen to the heterologous antibodies. The B cells may be reduced or eliminated by contacting the heterologous antibody with an anti-idiotypic antibody. Methods of making, and using the modified B cells for prophylaxis and therapy for a variety of conditions are provided. The B cells are modified at an IgH locus, an IgK locus, and combinations thereof. Modified B cells maintain allelic exclusion.

Abstract: Disclosed herein are expanded NK cells and methods of using thereof for treating, preventing, reducing, and/or inhibiting a microbial infection.

Abstract: The invention relates to immunogenic peptides comprising T-cell epitopes and new oxidoreductase motifs with increased activity, and their use in the treatment and/or prevention of type-1 diabetes (T1D), multiple sclerosis (MS), neuromyelitis optica (NMO), or rheumatoid arthritis (RA) in subjects.

Abstract: Provided is an antibody or an antigen-binding fragment thereof, a T cell antigen receptor, an immune cell expressing the T cell antigen receptor (TCR), and a preparation method therefor and the use thereof. The TCR can specifically recognize corresponding pMHC complexes, activate TCR T cells, and produce high-level cytokines IFN?, IL2, TNF?, significantly kill target cells and prolong the life of tumor-bearing mice.

Abstract: Anti-BCMA antibodies and chimeric antigen receptors (CARs) are provided. Immune cells expressing the anti-BCMA CAR can be used to treat cancer. The anti-BCMA antibodies and CARs can recognize the extracellular domains of human BCMA. The anti-BCMA CAR T cells show specific cytotoxicity towards BCMA-positive target cells.

Abstract: Provided herein are methods of producing engineered T cell compositions enriched for CD57 negative and/or CD27 positive T cells, such as from a plurality of donors. In some embodiments, the T cells are engineered with a recombinant receptor, such as a chimeric antigen receptor (CAR). Also provided herein are engineered T cell compositions containing T cells enriched for CD57 negative and/or CD27 positive T cells derived from a plurality of different donors, including compositions in which the T cells are engineered with or express a recombinant receptor (e.g. CAR). Also provided are methods of using the engineered T cell compositions in adoptive therapy, including in connection for cancer immunotherapy, such as for allogeneic therapies or for administration to one or more subjects in which the T cells are not derived from the subject(s) to whom the compositions are administered.

Abstract: Disclosed are T-cells that are positive for CD49f and which have an enhanced function compared to CD49f? cells. Methods of isolation of CD49f+ T-cells, as well as compositions and kits thereof are also disclosed. Additionally, enriched CD49f+T-cell populations have an increased proliferative potential, long-term survival and significantly improved efficacy in an adoptive therapeutic setting. The CD49f+ T-cells and CD49f+ T-cell enriched T-cell populations are useful in a range of applications, including for use in treating or inhibiting the development of diseases with immune dysfunction and methods of assessing risk of disease and potential responsiveness in immunotherapy. CD 19 CAR-T cells derived from CD49f+ T-cells and their use in a method of treatment of cancer is also disclosed.

Abstract: The present invention provides compositions and methods for inducing a CAR mediated trans-signal in a T cell. The trans-signaling CAR T cells comprise a first CAR having a first signaling module and a second CAR having a distinct second signaling module. The present invention also provides cells comprising a plurality of types of CARs, wherein the plurality of types of CARs participate in trans-signaling to induce T cell activation.

Abstract: The present disclosure provides methods of preparing immune cells, e.g., T cells and/or NK cells, comprising contacting the cells with programmable cell-signaling scaffolds in a medium comprising at least about 5 mM potassium ion. In some aspects, the methods disclosed herein increase the number of less-differentiated cells in the population of cells. In some aspects, the cultured cells are engineered, e.g., to comprise a chimeric antigen receptor (CAR) or an engineered T cell receptor (TCR). In some aspects, the cells are administered to a subject in need thereof.

Abstract: Disclosed herein are methods to improve effectiveness of cancer vaccines by increasing high affinity T cells number. Methods of eliciting an immune response in a treatment subject in need thereof and methods of treating cancer and/or a tumor in a subject are also disclosed.